News

In September 2016, Jerry Cahill and Emily Schaller joined a high-profile effort to persuade cystic fibrosis patients like themselves to cycle to improve their health. The title of the event said it all: It was the third annual Bike to Breathe adventure. Convinced that cycling helps them combat a disease…

The U.S. Food and Drug Administration (FDA) has approved Digestive Care’s Pertzye (pancrelipase) to be given to cystic fibrosis patients through a so-called G-tube — a feeding tube delivering nutrition directly to the stomach through a hole in the abdomen. Pertzye is an enzyme replacement therapy for exocrine pancreatic…

Three triple-combination treatments from Vertex Pharmaceuticals showed promising results in Phase 1 and Phase 2 clinical trials in cystic fibrosis (CF) patients with one F508del mutation and one so-called minimal function mutation (F508del/Min) in the CFTR gene, the defective gene that causes CF. Vertex announced that these are the first data to demonstrate…

Results of an online survey, conducted by the cystic fibrosis (CF) community GeneFo, showed that a main concern of CF patients is their intimate lives and sexuality. In an attempt to help CF patients and caregivers to find ways, alone or as a couple, to overcome the physical…

The Italian health insurance system has agreed to cover cystic fibrosis patients’ use of Vertex Pharmaceuticals‘ Orkambi (lumacaftor/ivacaftor). Those eligible for the Italian Medicines Agency coverage are patients 12 and older who have two copies of a particular mutation of the gene that is defective in the disease. That is the…

The U.S. Food and Drug Administration is promoting a targeted treatment approach known as precision medicine to help people with diseases stemming from specific, and often rare, genetic features. Its efforts include expanding the use of approved therapies to other genetic-based conditions, and pushing for the development of more biomarkers…

Synspira’s SNSP113 gives antibiotics more punch against the Burkholderia cepacia infections that cystic fibrosis patients experience, a study reports. The research, published in PLoS One, was titled “Novel glycopolymer sensitizes Burkholderia cepacia complex isolates from cystic fibrosis patients to tobramycin and meropenem.” Lung infections caused by Burkholderia cepacia are difficult to…

A first-grader with cystic fibrosis (CF), Conor Fink, inspired a close family friend to hop on his bicycle and spend his summer pedaling from the West to East Coast — Oregon to New York — to  raise money for Claire’s Place Foundation, a nonprofit helping children and families affected by the disease.

Anthera Pharmaceuticals has received news that could help it accelerate patient enrollment in a Phase 3 clinical trial of Sollpura (liprotamase) as a treatment for a digestive disorder known as cystic fibrosis-triggered exocrine pancreatic insufficiency. The news is that a key Cystic Fibrosis Foundation committee approved the company’s trial design. The sign-off…

A Phase 3 study of Sollpura (liprotamase), a non-porcine enzyme replacement therapy aiming to treat exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF) better than existing porcine-derived therapies, is now recruiting about 150 pediatric and adult patients in the United States, Europe and Israel. Specifically, the expanded trial will evaluate the efficacy — or non-inferiority — of Sollpura compared…